7:30 am Registration & Welcome Coffee
8:25 am Chair’s Opening Remarks
- Manmohan Singh Chief Technology Officer, Beam Therapeutics
8:30 am Lipid-Based Nanocarriers: Where Are We Today?
- Gaurav Sahay Associate Professor, Oregan State University
- As industry witnesses a diversification how lipid-based delivery vehicles, as new lipids are discovered, payloads loaded and diseases beyond infectious targeted, where is groundbreaking work taking place and laying the foundations for future innovation in drug delivery?
- An overview of industry’s progress and where further research will be critical to ensuring continued translation of next-generation delivery vehicles to the clinic and towards patients
Beyond the Liver: Achieving Efficacious Extrahepatic Delivery
9:00 am Nucleic Acid Delivery Systems for RNA therapy and Genome Editing
- Daniel Anderson Professor of Chemical Engineering and Institute for Medical Engineering & Science, Massachusetts Institute of Technology
- Development of new RNA delivery systems to non-hepatic tissue, including lung and HSC’s
- Advances in circular RNA delivery
- High throughput development of RNA delivery systems
9:30 am Polymer Lipid Hybrids: A Chemical Toolbox for LNP Formulations
- Vicent Nebot Chief Technology Officer & Head of EU, Curapath
- Novel polymer-lipids, beyond PEG
- Advantages of Polysarcosine PSar
- Lipid Manufacturing
- LNP Manufacturing
10:00 am Speed Networking Break
Meet and connect with your peers in this dedicated networking session
10:45 am The Future of Tissue-Targeted Lipid Nanoparticle Nucleic Acid Delivery
- Stephan Stern Laboratory Co-Director, Director of Research & Development, Nanotechnology Characterization Laboratory, National Cancer Institute
- Current marketed lipid nanoparticle-based nucleic acid therapeutics and future tissue targeted applications
- Nucleic acid lipid nanoparticle formulation and characterization, non-targeted LNP biodistribution, optimizing tissue targeted delivery
- Examples from: Hematopoietic stem cell-targeted CRISPR LNP for Sickle cell treatment ; Targeted siRNA delivery for pancreatic cancer therapy– in vivo models and translational development ; APC-targeted mRNA LNP vaccines for increased potency and safety
11:15 am Fireside Chat: Lipid Design Principals: How Does Nanoparticle Design & Delivery Route Influence Extrahepatic Targeting Properties?
- Whitney Shatz-Binder Principal Scientist, Genentech
- Justin Cohen Director, Molecular Discovery, Analytical Conjugation Sciences, Pfizer
- How to better engineer and select particles with the most attractive properties?
- Which novel lipid discoveries are showing promise for efficient in vivo delivery of nucleic acid drugs?
- How can we better characterize lipids and define their properties and chemical identities to facilitate the next-generation development of lipids?
- What formulation considerations are needed for differing routes of delivery, for instance intramuscular, subcutaneous?
12:00 pm Reserved for Particle Works
12:15 pm Lunch Break
Take this chance to meet the expert speakers, connect with your peers and explore our exhibition booths
1:30 pm Extrahepatic Delivery of mRNA Based on Ionizable Cationic Lipid Library: From Hepatocyte to Activated Stellate Cells, from Normal to Disease State
- Hideyoshi Harashima Professor of Pharmaceutics and the chair of the Laboratory of Molecular Design of Pharmaceutics, Faculty of Pharmaceutical, Hokkaido University
- From in vivo screening, we have identified a few ionizable cationic lipid such as CL4H6 which can exert silencing activity of siRNA more efficient than MC3 in hepatocytes after intravenous administration
- CL4H6LNP can be applied for genome editing successfully, we established to formulate CRISPR/Cas9 ribonucleoprotein (RNP) into LNP
- Then we challenged to find a new system which can target an activated stellate cells (aHSC) which play an important role in the progression of liver fibrosis based on our library of iCL; We have identified CL15A6 which has different hydrophilic head group and hydrophobic lipid tail from CL4H6
- Another example is an optimized formulation to target splenic B-cells for pDNA, which can induce efficient antitumor effects via enhanced CTL activity
- These results indicate that a tailored formulation with ionizable cationic lipids library can provide a cell selective targeting of nucleic acids/RNP without ligands for next generation nanomedicine
2:00 pm Reserved for Metis Therapeutics
Solidifying Safety for Next-Generation Hepatic Delivery
2:15 pm Delivering Innovative Gene Editing Therapies to The Liver in Addition to Extrahepatic Locations with Optimized Selectivity
- Gulen Yesilbag Tonga Senior Scientist, Delivery Sciences, Tessera Therapeutics
- Tessera’s Gene Writer™ systems, which can be delivered as RNA or DNA, enable the correction of single nucleotides, the deletion or insertion of short DNA sequences, and the writing of exons or entire genes into the genome
- Proprietary non-viral lipid nanoparticles are designed to deliver Gene Writers™ to targeted tissues, potentially enabling the application of Gene Writing™ therapies in vivo
- Presentation highlighting improved safety and efficacy of cell therapy delivery for hepatic and extrahepatic delivery in vivo
2:45 pm Afternoon Break
Grab a coffee and explore our exhibition booths, networking and more!
Payloads Navigating Complex Biological Barriers
- Devyn Smith Chief Executive Officer, Arbor Biotech
3:15 pm Interactive Roundable: Enabling New Modalities to Achieve Their Therapeutic Potential
- Manuel Sanchez-Felix Senior Fellow, Novel Delivery Technologies, Novartis
- Saikat Manna Senior Scientist, Delivery & Formulation, Sanofi U.S.
As our understanding of new nucleic-acid modalities improve, and bioavailability assessments reveal a necessitated need for drug delivery vehicles, there is urgent need for lipid-based nanoparticle delivery system development to keep up with pharmaceutical progress to see the therapeutic potential of pipelines reached.
With LNPs established for mRNA / RNA therapies, and new lipid chemistries being discovered, use this roundtable discussion to gain peer-to-peer feedback:
- Could a greater understanding of barriers related to targeting different tissues allow for a better class of LNPs be developed? Considering route of administration and tissue related barriers
- What critical carrier attributes need to be addressed in order protect payloads from release when traveling across membranes?
4:15 pm Reaching the Nucleus: Lipid-Based DNA Therapeutics
- Badri Ananthanarayanan Head of Delivery, EARLI
- The final frontier in gene delivery: nuclear delivery of DNA. How do LNPs stack up against older platforms?
- How can we systematically engineer nanoparticles for improved nuclear delivery of DNA?
- In vivo validation to develop translatable platforms, with rigorous safety examinations supporting clinical translation
4:45 pm Chair’s Closing Remarks & End of Day One
- Devyn Smith Chief Executive Officer, Arbor Biotech
5:15 pm Poster & Networking Session
The learning and networking continues! This is the perfect opportunity to present & discuss your recent work with fellow drug delivery leaders & innovators!